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Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

Description

The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A\>G (p.Cys998X) mutation in the CEP290.

Conditions

Leber Congenital Amaurosis 10BlindnessLeber Congenital AmaurosisSensation DisordersVision DisorderNeurological ManifestationsEye Diseases, HereditaryEye DiseasesEye Disorders CongenitalRetinal Disease
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Related Conditions:

Leber Congenital Amaurosis 10BlindnessLeber Congenital AmaurosisSensation DisordersVision DisorderNeurological ManifestationsEye Diseases, HereditaryEye DiseasesEye Disorders CongenitalRetinal Disease

Study Overview

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Study Details

Study overview

The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A\>G (p.Cys998X) mutation in the CEP290.

A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 Gene

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

Condition
Leber Congenital Amaurosis 10
Intervention / Treatment

-

Contacts and Locations

Minneapolis

University of Minnesota Medical School, Minneapolis, Minnesota, United States, 55455

Philadelphia

University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics, Philadelphia, Pennsylvania, United States, 19104

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. Confirmed clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A\>G mutation in CEP290.
  • 2. Adults: \>=18 years / Minors: 6 to \<18 years.
  • 3. BCVA (FrACT) equal to or worse than logMAR +0.4 (approximate Snellen equivalent 20/50) to +2.9 logMAR based on quantifiable, reliable FrACT. LP subjects with documented evidence of prior better vision eligible.
  • 4. Symmetrical disease between the two eyes as defined by a BCVA (FrACT) within 0.2 logMAR at baseline.
  • 5. Detectable ONL in the macular area as determined by the CRC at Screening.
  • 1. Mutations in genes other than the CEP290 gene associated with other IRD diseases or syndromes.
  • 2. Presence of any ocular pathology in either eye that may make comparison of the eyes not feasible.
  • 3. Presence of unstable concurrent CME, or subject started on (or changed dose of) topical or systemic carbonic anhydrase inhibitor treatment in the 3 months prior to enrollment. CME is allowed if stable for 3 months (with or without treatment).
  • 4. Presence of any clinically significant lens opacities/cataracts based on the AREDS lens grading scale.
  • 5. Any prior receipt of genetic or stem-cell therapy for ocular or non-ocular disease.

Ages Eligible for Study

6 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Laboratoires Thea,

Study Record Dates

2028-10